A - cyclophosphamide/antithymocyte globulin
B - cyclophosphamide/rituximab (verbal communication Dr. Federenko, Moscow, Russia)
C - split dose cyclophosphamide/ rituximab
G-CSF = granulocyte colony-stimulating factor,
HSC = hematopoietic stem cell,
rATG = rabbit anti-thymocyte globulin.
Uppsala (Uppsala, Sweden) and the other from Northwestern University (Chicago, US).
Both studies demonstrated dramatic improvements in neurologic disability of a magnitude that had never previously been reported in clinical trials with DMTs.
Since then several studies of HSCT performed predominately or solely for RRMS have been published with similar results (Table 41.1).
To better understand the profound effect of HSCT for MS, the data were collected before some of the highly active DMTs were licensed, and being a real world setting, a percentage of patients could have been off treatment for variable periods of time.
In comparison, after HSCT for RRMS, NEDA is roughly 70-90% at 2 years and 68-80% at 5 years
NEDA, while a useful clinical endpoint, does not capture improvement in neurologic disability that is most often measured